Amicus Therapeutics - Earnings Call - Q2 2025

Executive Summary

• FOLD delivered Q2 2025 revenue of $154.7M, +22% reported YoY (+18% CER), with Galafold $128.9M (+16% reported) and Pombiliti + Opfolda $25.8M (+63% reported). Currency was a ~$5M tailwind; ex-US represented ~58% of revenue.
• Results exceeded S&P Global consensus: revenue $154.7M vs $146.7M (+$8.0M, +5.5%)* and non-GAAP EPS $0.01 vs -$0.12*, while GAAP EPS was -$0.08 (non-GAAP beat driven by stronger top line despite $30M DMX-200 upfront in opex)*.
• Guidance reiterated: FY25 total revenue growth +15–22% CER; Galafold +10–15% CER; Pombiliti + Opfolda +50–65% CER; gross margin mid-80s; non-GAAP opex $380–$400M (incl. $30M DMX-200 upfront); positive GAAP net income in H2 2025.
• Strategic catalysts: Japan approval for Pombiliti + Opfolda (June), acceleration in new EU launches (Netherlands preferred therapy), and DMX-200 Phase 3 enrollment on track with FDA alignment on proteinuria endpoint; management reiterated a path to >$1B revenue by 2028.

What Went Well and What Went Wrong

What Went Well

• Strong top line and segment momentum: Q2 revenue +22% reported (Galafold +16% reported; Pombiliti + Opfolda +63% reported), with ex-US 58% mix and currency tailwind aiding reported growth.
• Geographic and access wins for Pombiliti + Opfolda: regulatory approval in Japan; Netherlands selected as preferred treatment; six countries recorded first patient starts in H1 2025; up to 10 new launch countries in 2025.
• Management conviction and execution: “seventeenth consecutive quarter of double-digit gains at CER,” reiteration of H2 GAAP profitability; CEO: “firmly positioned to reach GAAP profitability in the second half of 2025”.

What Went Wrong

• Higher operating expenses: GAAP opex +48% YoY to $148.9M (non-GAAP +56% to $127.8M), including the $30M DMX-200 U.S. licensing upfront.
• GAAP EPS negative: -$0.08 vs -$0.05 YoY, despite non-GAAP profitability; CFO noted GAAP would have been positive excluding the $30M payment.
• Sequential non-GAAP EPS softness: $0.01 in Q2 vs $0.03 in Q1 as the company absorbed higher opex and began transitioning to recognizing Pombiliti + Opfolda COGS in H2, with gross margin guided to mid-80s for FY25.

Transcript

Operator (participant)

Good morning, ladies and gentlemen, and welcome to the Amicus Therapeutics Second Quarter 2025 Financial Results Conference Call and Webcast. At this time, all participants are in a listen-only mode. Later, we will conduct a question-and-answer session, and instructions will follow at that time. As a reminder, this conference call is being recorded. I would now like to turn the conference over to your host, Mr. Andrew Faughnan, Vice President of Investor Relations. You may begin.

Andrew Faughnan (VP of Investor Relations)

Good morning. Thank you for joining our conference call to discuss Amicus Therapeutics second quarter 2025 financial results and corporate highlights. Leading today's call, we have Bradley Campbell, President and Chief Executive Officer, Sébastien Martel, Chief Business Officer, Dr. Jeff Castelli, Chief Development Officer, and Simon Harford, Chief Financial Officer. Joining for Q&A is Ellen Rosenberg, Chief Legal Officer. As referenced on slide two of the presentation, I would like to remind you that we will be making forward-looking statements on today's call. I encourage you to read the disclaimers in our slide presentation, the press release we issued this morning, and the disclosures in our SEC filings, which are all available on the IR portion of our corporate website. Forward-looking statements are subject to substantial risks and uncertainties, speak only as of the call's original date, and we undertake no obligation to update or revise any of the statements.

Additionally, you are cautioned not to place undue reliance on any forward-looking statements. At this time, it is my pleasure to turn the call over to Bradley Campbell, President and Chief Executive Officer. Bradley.

Bradley Campbell (President and CEO)

Great. Thank you, Andrew, and welcome everyone to our second quarter conference call. I'm very pleased today to highlight our strong quarter and positive outlook for Amicus for the remainder of the year. First, we've delivered yet another quarter of strong double-digit revenue growth on our core business in Pompe and Fabry disease. This is our 17th consecutive quarter with double-digit sales growth at CER, and we see that trend continuing for years to come. Second, we remain highly confident in our growth trajectory that you'll see throughout the remainder of the year. Galafold delivered 13% year-over-year patient growth this quarter and is on track to achieve the highest number of global patient starts this year.

For Pombiliti and Opfolda, Q2 marked the strongest quarter for commercial demand since our launch, with significant momentum both in the United States and the five new launch countries in Europe, as well as the existing launch countries there as well. Third, we're steadily building the body of evidence highlighting the differentiation of Pombiliti and Opfolda in Pompe disease, including a recent publication in muscle and nerve, which demonstrates the benefits for switching ERT experienced patients to Pombiliti and Opfolda. Fourth, we are reiterating our confidence that these two products will reach combined sales of $1 billion by the end of 2028. We continue to believe that Galafold is uniquely positioned to further its reach as diagnostic rates and patient access continue to improve, offering a substantial runway for sustainable growth, and Pombiliti and Opfolda becoming an increasingly meaningful contributor to our long-term performance.

As a reminder, each of these products has a $1 billion plus in peak sales potential. Fifth, we continue to advance our strategic partnership with Dimerix and DMX-200, a first-in-class compound in late-stage phase III development for a rare and life-threatening kidney disease. The ACTION3 pivotal study remains on track for full enrollment by the end of the year, marking a key milestone in this important program. DMX-200 and our opportunity to meet the significant unmet needs that exist for people living with FSGS is an important new part of the Amicus story, and we look forward to telling you more over the course of the year. Finally, as we continue to maintain our financial discipline, we reiterate that we are on track to achieve GAAP profitability in the second half of this year.

Altogether, we're pleased with our accomplishments this quarter and believe Amicus is in a very strong position to generate meaningful value for our shareholders and deliver on our mission for patients in 2025 and in the years ahead. With that, let me now hand the call over to Sébastien to go through some more detail. Sébastien?

Sébastien Martel (Chief Business Officer)

Thank you, Bradley, and good morning, everybody. Let's start with Galafold on slide five. You see that revenue reached $128.9 million this quarter, up 12% at constant exchange rates and up 16% in reported terms. The underlying growth of this product remains very positive and is driven by the number of new patient starts globally. We ended the quarter with more than 69% of the global market share for treated Fabry patients with amenable mutations. Galafold is clearly positioned as the treatment of choice amongst prescribers, and there are still many more potential patients eligible for our therapy. Turning to slide six, our leading markets continue to be the biggest driver of strong patient demand for Galafold. The U.S. actually contributed significantly to growth, reaching more than 1,000 PRS since launch, a major milestone.

When we look at the global mix of patients on Galafold today, which is about 65% naive and 35% switch, we're now seeing stronger uptake in naive populations. While we continue to achieve high market shares in countries where we've been approved the longest, there's still plenty of opportunity to switch patients over to Galafold and to keep growing the markets as we penetrate the diagnosed, untreated, and newly diagnosed segments. With underlying growth in patient demand at 13% this quarter and our projection of a record level of new patient starts this year, we remain highly confident in our full-year 2025 growth guidance for Galafold.

The key drivers behind the robust demand for Galafold, which we expect to continue well beyond 2025, are the following: first, finding new patients and penetrating into the diagnosed, untreated population, including shortening the pathway to diagnosis; second, expanding Galafold into new markets and extending the label; third, driving Galafold's share of treated amenable patients. We're actually seeing in our most mature market that we can reach 85%, 90% share, so we know that there's the potential to reach those levels globally. Fourth, sustaining compliance and adherence rates above 90% so that patients who go on Galafold predominantly stay on Galafold. On slide seven, we highlight the significant unmet need in Fabry disease today. Over 12,000 people receive Fabry treatment worldwide, while 6,000 diagnosed patients remain untreated.

Literature suggests actual prevalence makes it over 100,000 individuals, indicating a meaningfully larger underdiagnosed population than originally believed, and substantial market opportunity for Galafold. We're highly confident that a small molecule is a compelling treatment option for the untreated and undiagnosed populations, as indicated by the record high growth in naive new patient starts. We're just only scratching the surface with Galafold today. As disease awareness and enhanced diagnostics initiatives further shape the Fabry market, we're confident in the long-term potential for this medicine, which we think continues to be underappreciated. With excellent momentum, a sizable untreated population, and our strong IP protection, Galafold has a long runway well into the next decade and a clear path to surpassing $1 billion in revenue. Turning now to Pompe disease on slide nine, we outline our global launch progress with Pombiliti and Opfolda.

The second quarter revenue reached $25.8 million, up 58% at constant exchange rates. The majority of sales came from our initial five launch countries: the U.S., UK, Germany, Spain, and Austria, although, as I'll highlight in a moment, we launched into five new markets in Q2 alone. The U.S. represented approximately 42% of revenue, while ex-U.S. represented 58% of revenue. Q2 showed strong sales growth, as well as record levels of patient demand. We continue to see patients switching proportionally based on market shares, as well as a broadening and deepening of prescriptions, with more sites coming online and multiple new prescriptions from physicians. Given these indicators, we are reiterating our full-year 2025 revenue growth guidance for Pombiliti and Opfolda, of 50%-65% growth at constant exchange rates.

Our guidance implies a healthy exit rate heading into the next year, and we remain highly confident in the 2025 and long-term outlook for this therapy. We expect Pombiliti and Opfolda to be a major contributor to multi-year growth for Amicus, based on key growth drivers, namely continuing to increase the number of net new patients, increasing the depth and breadth of prescribers, launching in new countries, including up to 10 this year alone, differentiating our therapy through evidence generation and real-world evidence, and last, maintaining 90% plus compliance and adherence rates. Moving to slide 10, looking at the geographic expansion of Pombiliti and Opfolda. In the second quarter, we recorded revenue in 11 countries. Six countries have their first patient starts during the first half of 2025: Italy, Switzerland, Portugal, the Czech Republic, Sweden, and the Netherlands.

We're very pleased that Pombiliti and Opfolda were selected as a preferred treatment for adults with LOPD in the Netherlands. It's an important market. All patients go through one site, and the site is actively transitioning patients. It will be a key driver for us in the second half of this year. We estimate well over 100 patients and intend to take up to 70% of this population. This will then become the largest cohort in any single center worldwide and definitely a rich source of data on Pombiliti and Opfolda. We also recently received regulatory approval in Japan and are excited to have a label indicated for people ages 15 and older. We're also continuing our work to secure broad patient access throughout the EU.

I hope that the commercial overview provides a strong sense of the continued execution and growth in Galafold and the building momentum in the launch of Pombiliti and Opfolda. With that, I will now hand the call over to Jeff to highlight the work we do to further differentiate Pombiliti and Opfolda. Jeff?

Jeff Castelli (Chief Development Officer)

Thank you, Sébastien, and good morning, everyone. Starting on slide 11, we highlight a few examples of our rapidly expanding and diverse body of evidence supporting the differentiation of Pombiliti and Opfolda. In Pompe disease, and specifically on slide 12, we summarize two recent case studies recently presented at the ACMG 2025 annual meeting, supporting the experiences of individuals switching from Nexviazyme to Pombiliti and Opfolda. These case studies, along with the growing body of real-world evidence, continue to show that improvement is possible for many patients when switching to Pombiliti and Opfolda. We believe our ongoing efforts to grow this body of evidence will ultimately drive wider adoption of Pombiliti and Opfolda. Moving to slide 14, as previously announced, we took a major step forward in our strategy to strengthen our portfolio through a very successful U.S.

licensing agreement with Dimerix to commercialize DMX-200, a first-in-class treatment in late-stage development for FSGS, a rare and potentially fatal kidney disease. With blockbuster market potential, we remain highly encouraged by the data seen to date and believe this asset brings immediate strategic value to Amicus and will create value for patients and for shareholders. Moving to slide 15, we think it's important to highlight the very differentiated and very compelling mechanism of action of DMX-200, which continues to resonate well with physicians and the FSGS research community. There are currently no FDA-approved therapies for FSGS. Standard of care includes nonspecific therapies such as corticosteroids, calcineurin inhibitors, and angiotensin receptor blockers, none of which adequately address the monocyte-driven inflammatory aspect of FSGS.

DMX-200 is an oral small molecule taken in combination with ARBs that specifically target this monocyte-driven inflammatory component of FSGS by inhibiting signaling from the angiotensin I receptor in chemokine receptor type II heterodimer that is formed in damaged kidney cells. It delivers a kidney-selective anti-inflammatory effect directly targeting this key unaddressed driver of disease, in particular in patients, which are many of them in FSGS, with persistent proteinuria and active inflammation. Preclinical and phase II studies support this mechanism of action and demonstrate impacts on proteinuria with a well-tolerated safety profile to date, with no evidence, importantly, of the MCP-1 rebound effects observed with traditional CCR2 inhibitors. Moving on to slide 16, we are very impressed by the strong momentum Dimerix has built and the growing body of evidence supporting the transformative potential of DMX-200.

The pivotal phase ACTION3 trial is progressing really well, with more than 75% of patients now enrolled and remains on track for full enrollment by year-end. The study is robustly designed and strongly powered, with several successful interim analyses completed to date. Importantly, there is FDA alignment on proteinuria as the primary endpoint for approval. Taken together with all these facts, we believe DMX-200 is positioned to truly be a meaningful advancement for FSGS patients. Following additional analysis and coordination with the PARASOL Consortium over the coming months, we anticipate requesting an additional meeting with the FDA to discuss the next interim assessment of efficacy from the ACTION3 study and next steps for DMX-200. With that, let me now hand the call over to Simon to review our financial results and outlook. Simon?

Simon Harford (CFO)

Thank you, Jeff. Our financial summary begins on slide 18 with our income statement for the second quarter ending June the 30 of 2025. For Q2, we achieved total revenue of $154.7 million, which is a 22% increase over the same period in 2024. At constant exchange rates, revenue grew 18%. The global geographic breakdown of total revenue in the quarter consisted of $90.4 million, or 58% of revenue generated outside the U.S., and the remaining $64.3 million, or 42% coming from the U.S. Cost of goods sold as a percentage of net sales was 10% for Q2 as compared to 9% in the same period last year. Total GAAP operating expenses increased to $148.9 million for the second quarter of 2025 as compared to $100.4 million in the second quarter of 2024, an increase of 48%.

It is important to remember that Q2 operating expenses included the upfront payment of $30 million for the U.S. licensing rights to DMX-200. On a non-GAAP basis, total operating expenses increased to $127.8 million for the second quarter as compared to $82.1 million in the second quarter of 2024, an increase of 56%. We define non-GAAP operating expense as research and development and SG&A expenses, excluding stock-based compensation expense, loss on impairment of assets, changes in fair value of contingent consideration, restructuring charges, and depreciation and amortization. On a GAAP basis, net loss in the second quarter of 2025 was $24.4 million, or $0.08 per share, compared to a net loss of $15.7 million, or $0.05 per share for the second quarter of 2024. Excluding the $30 million upfront payment related to the DMX-200 agreement, we would have delivered positive GAAP net income for the quarter.

In Q2 2025, non-GAAP net income was $1.9 million, or $0.01 per share, compared to non-GAAP net income of $18.5 million, or $0.06 per share in the second quarter of 2024. Cash equivalents and marketable securities were $231 million as of June 30, 2025, compared to $250 million as of December 31, 2024. On slide 19, we are reiterating our full-year financial guidance for 2025 as follows: total revenue growth of 15%-22%, Galafold revenue growth of 10%-15%, Pombiliti and Opfolda revenue growth of 50%-65%. All of these growth rates are at constant exchange rates. Gross margin is expected to be in the mid-80s. Non-GAAP operating expense guidance remains at $380 million-$400 million, and we anticipate positive GAAP net income during the second half of 2025.

As mentioned earlier this year, 2025 will be a hybrid year for Pombiliti and Opfolda COGS, as we have worked through the previously expensed or zero-cost inventory during the first half of the year. As a result, we expect our gross margin to be in the mid-80s for 2025, as we begin to recognize Pombiliti and Opfolda COGS through the P&L in the second half of the year. With that, let me turn the call back over to Bradley for our closing comments.

Bradley Campbell (President and CEO)

Great. Thank you, Simon, Jeff, and Sébastien. As we come to the end of our presentation, here's just a quick reminder of our strategic priorities for the year. In closing, I want to reiterate how encouraged we are by the growing demand for our therapies and the very promising phase III asset that we've added to our pipeline. We see a clear path to sustained growth in 2025 and beyond, and we've demonstrated that we have the portfolio and capabilities to deliver that at a highly attractive growth trajectory. Amicus continues to represent a very differentiated company in biotech and rare disease, with now 17 successive quarters of double-digit revenue growth, a de-risked portfolio in growing categories, and an efficient and highly effective organization that is laser-focused on delivering for patients with rare diseases.

I have full confidence that we will continue to advance transformative treatments and create lasting value for patients and shareholders alike. With that, operator, we can now open the call to questions.

Operator (participant)

Ladies and gentlemen, if you have a question, please press star one one on your touch-tone telephone. At this time, we request that you only ask one question. If you have an additional question, please enter back into the queue. Thank you. Please stand by while we compile the Q&A roster. Our first question comes from the line of Anupam Rama of JPMorgan. Your line is now open.

Priyanka Grover (Analyst)

Hi, guys. This is Priyanka on for Anupam. Congrats on the quarter. Looking at the real-world evidence, what clinical assessments really resonate with physicians and KOLs when patients switch from Nexviazyme to PomOp? Are there differences between the U.S. and OUS? Thanks.

Bradley Campbell (President and CEO)

Thanks, Priyanka. I'll turn it over to Jeff in a minute to provide some detail. I think the really important thing here is now that we have multiple treatments, this is exactly the question that I think we're helping to drive in the scientific community. As we develop more evidence and as we demonstrate the effects of Pombiliti and Opfolda, I think that will continue to be an important part of the story. Jeff, maybe talk kind of how that's evolved somewhat with these new therapies and what the physicians and patients are looking at.

Jeff Castelli (Chief Development Officer)

Yeah, thanks for the question. The physicians are looking for when switching from Lumizyme to Pombiliti are not that different from what they're looking for when they're switching from Nexviazyme to PomOp. I mean, the majority of patients that are switching here early in the launch tend to be those that are on Nexviazyme, have either were naive and went on Nexviazyme or switched from Lumizyme and are not having the outcome that they had hoped when they went on Nexviazyme. They're looking for either stability of its declining function or improvements in things where there had been stability previously. Typically, as shown on the slide in the presentation, they look at things like biomarkers, muscle strength, and then things like six-minute walk, FVC, as well, of course, as just quality of life. How is the patient doing day-to-day and activities of daily living?

What was really exciting from the two case studies highlighted here in the presentation, as well as what we're hearing more broadly, is that similar to what we saw in trials and so far in some of the different studies ongoing, those patients switching from Nexviazyme seem to also be having, on average or in many cases, a very positive experience on that switch. It really is not that different switching from Nexviazyme than switching from Lumizyme. It's a pretty similar process.

Bradley Campbell (President and CEO)

Thanks, Jeff. I don't think to your question, Priyanka, I do not think there's really much difference between the U.S. and other geographies.

Operator (participant)

One moment for our next question. Our next question comes from the line of Joe Schwartz of Leerink. Your line is now open.

Joe Schwartz (Analyst)

Great. Thanks very much, and congrats on a strong quarter. My one question is, I guess, will be on with tariffs and MFN remaining a topic of discussion now. I was wondering if you could just update us on your additional manufacturing facility for PomOp in Ireland. When could that come online? Does that get you to where you think you need to be to the extent anyone can forecast the future in this regard? Do you think that could supply all of the PomOp that you forecast you'll need? Can you just remind us how much drug you've stockpiled in the U.S.? Thanks.

Bradley Campbell (President and CEO)

Thank you very much, Joe. A few questions there. Maybe I'll kind of go in reverse order. We brought all of the material into the United States for Pombiliti and Opfolda that we needed for commercial use this year and clinical use as well. That is what led us to say that there is no material impact of tariffs on our P&L this year. Any forecast that we've been able to do going forward, even at relatively conservative levels, is very manageable within our P&L based on the new global supply strategy. As it relates to Ireland, from a capacity perspective, yes, especially as we look towards the second-generation manufacturing process, which will evolve over the next kind of five years, that could supply the global demand that we forecast. It is very likely that we may have a secondary site, just from a good strategic perspective.

Right now, that site is China, which could serve Europe and ex-U.S. markets, but there might be other opportunities there. I would point you to the announcement we had in Q1, which was for the very first time bringing drug product manufacturing to the United States with a collaboration with Sharp. We may continue to evolve that as time goes on, depending on the political landscape. I think we've been very prudent and very forward-looking to have a diverse supply chain. The last question in terms of when those things will come on board. For Ireland, we believe that that material will enter the commercial supply chain towards the back half of this year in Europe and then sometime next year in the United States, which is exactly what we forecasted. We think we're in really good shape.

We've been able to navigate all the kind of headwinds and challenges that are out there, and we expect a very robust, optimized supply chain going forward.

Joe Schwartz (Analyst)

Very helpful. Thanks for the insight.

Bradley Campbell (President and CEO)

Yeah, thanks, Joe.

Operator (participant)

One moment for our next question. Our next question comes from the line of Maxwell Skor of Morgan Stanley. Your line is now open.

Maxwell Skor (Analyst)

Great. Thank you for taking my question and congratulations on the quarter. Now that you've read the brief submitted by Aurobindo for summary judgment, do you still feel confident in your IP position and the potential for a settlement? Thank you.

Bradley Campbell (President and CEO)

Yeah, thanks, Max. Just as a reminder, we've said that we remain highly confident in the strength of our IP and the long-term opportunity to support Fabry patients in the many years to come. Of course, the settlement with Teva reinforces our confidence in the strength of our case against any remaining litigants, including Aurobindo, and overall the strength, breadth, and depth of our IP estate. Because we're still in litigation, we can't comment further on the details there. I would just say we remain highly confident that our IP is long and is supported. We would remind everybody, just statistically, the vast majority of these cases ultimately lead to settlement, in particular when one party reaches a settlement first. Hopefully, that's helpful, and I look forward to providing further updates as time goes on.

Maxwell Skor (Analyst)

Great, thank you.

Operator (participant)

One moment for our next question. Our next question comes from the line of Ritu Baral of TD Cowen. Your line is now open.

Joshua Fleishman (Analyst)

Hi, Brad. This is Joshua Fleishman on the line for Ritu. One multipart question. How are timelines progressing for the new U.S. manufacturing process, and what do you think its impacts could be on COGS? How do you view additional pipeline expansion, and what would your priorities be? What should we expect for Pombiliti's launch in the next 12 months as more next biosine patients approach the important two-year mark for treatment reevaluation? How is the current reimbursement situation? Thank you.

Bradley Campbell (President and CEO)

I see you've adopted Ritu's approach to one question with multiple parts. We'll do our best to answer all of them. Thank you, Joshua. Maybe Sébastien, do you want to speak to the U.S. drug product manufacturing facility and the general timelines there? We haven't given real specifics, but just a flavor for sort of how that will evolve, and then we'll take the next few as well.

Sébastien Martel (Chief Business Officer)

Yes. Thanks, Brad. As you know, you saw that as we announced in Q1, we signed an agreement with Sharp Sterile to bring the Pombiliti drug product manufacturing to the U.S., essentially onshoring DP manufacturing for Pombiliti. We'll be working through our PPQs in the next few quarters. We haven't shared specific timelines yet on when that site might be up and running. In the meantime, you've heard from Brad the progress we've made on the DS side of things from the Dundalk Ireland site. I'm very excited for the progress we're making here with both EMA and FDA. In parallel, we have another site for DP in Germany, Leverkusen, where we're also making great progress and actually have just started PPQ runs as we speak. We're very advanced in our overall manufacturing strategy for Pombiliti.

Bradley Campbell (President and CEO)

Thanks, Sébastien. Just to hit a couple of the other points you made there, I'll start with the maybe quickest one first. Reimbursement continues to go really well in all of our geographies. You've seen we have oftentimes been firsts or fastest in multiple markets, like the first-ever approval from NICE prior to MHRA approval, fastest to getting to reimbursement in a number of markets, getting lead position in a number of markets like the Netherlands, which Sébastien highlighted. I think that just reflects our approach to maximizing access and delivering value for all stakeholders, and we'll continue to do that. To your point about Nexviazyme switches, as we've said previously, a significant portion in the United States, as an example of the Nexviazyme community, have come to that sort of two-year switch point, and that obviously will continue to grow over time.

I think it was important, Sébastien, to highlight that we are switching sort of relative to market share. In the U.S., where the majority of patients are on Nexviazyme, a majority of our new patients are coming from Nexviazyme as well. Your last question on pipeline expansion, we're really excited about DMX-200 and about telling that story in more detail over the course of the year. From a BD perspective, we still think there are opportunities to leverage our infrastructure and capabilities globally, and we continue to be focused on late-stage de-risked assets, near commercial assets, similar to the DMX-200. Hopefully, that was helpful. I think I caught them all. Thanks, Joshua, for the questions.

Joshua Fleishman (Analyst)

Yeah, thank you so much.

Operator (participant)

One moment for our next question. Our next question comes from the line of Eliana Merle of UBS. Your line is now open.

Tejas Wein (Analyst)

Hi, this is Tejas on for Eli. Congrats on the quarter. Could you guys give a little bit more color on how starts are going in ex-U.S. markets? I know you've mentioned the Netherlands, Sweden. Any color there would be great. Thank you.

Bradley Campbell (President and CEO)

Sébastien, you want to just give a few highlights on some of the exciting things we're seeing in some of the different markets? There's lots that we could tell, but maybe Spain would be a good one to highlight. I don't know, Germany, UK, etc.

Sébastien Martel (Chief Business Officer)

Yeah. Yeah. You know, we continue to see strengths from the first market we launched, as Brad mentioned, the UK and Germany. Interestingly, in the UK, when you take into account the EAMS program and the fact that Pomb was essentially available for physicians almost two years prior to launch, we've been in that market for about three and a half years, and now our market share is reaching 35%. We see that market with a lot of enthusiasm as to what we could achieve in other markets as well. Germany remains strong. Spain, as you said, Brad, this was an interesting situation where we actually launched neck and neck with Nexviazyme, and we're seeing, again, make significant inroads from a market share standpoint in that market.

We've got smaller markets like Sweden, where PomOp is actually the drug to be on if you have LOPD in Sweden, and we have a disproportionate market share in the Swedish market as a result of that. We're launching in Italy as we speak. I did say that this year alone, we have now six new countries in which we launched, and we anticipate another four for the remaining of the year. That would include our Japanese launch in the second half. Lots of room to continue to grow Pombiliti and Opfolda.

Bradley Campbell (President and CEO)

Great. Thanks, Sébastien. Thanks for the question.

Operator (participant)

One moment for our next question. Our next question comes from the line of Kristen Kluska of Cantor Fitzgerald. Your line is now open.

Kristen Kluska (Analyst)

Hi, good morning, and congrats on the nice revenue beat. For PomOp, with 40% of the patient pool treated on next Nexviazyme reaching that two years this year, curious now that you have more data behind your hands, what's making patients switch right at two years versus earlier versus perhaps later on? Is it their total time diagnosed with Pompe? Are there any specific drivers that, again, would make someone switch earlier, later, or right at that two-year mark? Thank you.

Bradley Campbell (President and CEO)

Yeah, thanks, Kristen. I think embedded in your question is the reality, which is it's not like at two years, everybody switches. It is a continuum. I think the earlier switches have been people who were clearly declining on, regardless of what therapy they're on, Lumizyme or Nexviazyme, and I think that will continue to happen. If it's an obvious decline, I think physicians and patients are looking for something new and different. That probably skews the initial patient population also to a more severe patient population. Sometimes that can be an older patient population. The exciting thing is we're the only product in our head-to-head study that showed an improvement, and I think that's, as Jeff said, improvement is possible with this product. I think that's really the promise of what we can offer. Over time, though, I think two things can happen.

The first is what Jeff talked about earlier, which is I think the physician community now, I don't think, I know, is asking themselves, "What do we need to look at? How closely do we need to look at some of these measures to find a more subtle early predictor of decline?" I think that's a very important conversation that's happening right now. The holy grail in what our ambition is, and we kind of saw this with Galafold, is instead of waiting for decline, eventually, we believe we can establish Pombiliti as the best therapy out there, and then you'll see a proactive switch. The other dynamic that's kind of flowing through all of that is also where patients sort of raise their hand and say, "Hey, I'm not feeling well," or, "I want to try something new," and that's been an exciting part of the story as well.

Hopefully, that gives you a flavor of some of the dynamics that you're getting at in your question.

Jeff Castelli (Chief Development Officer)

Brad, the only other thing I'd add is just looking at the long-term data that we've seen from Nexviazyme, from Lumizyme, for example, what you see on that kind of average response across parameters like six-minute walk, FVC, is that after one year, two years, you generally see on average a continued slow decline. You would expect, just thinking about people not doing well, there's going to be more patients not doing well after three years of Nex than after two or after four years of Nex versus three. That will continue to add people if you're just looking at those not doing well. We'd expect there to be a kind of continued growth of that over time.

Operator (participant)

Thank you. One moment for our next question. Our next question comes from the line of Dennis Ding of Jefferies. Your line is now open.

Dennis Ding (Analyst)

Hi. Good morning. Thanks for taking our questions. Two, for me, on FSGS, can you go into a little bit more detail on the regulatory alignment you have with the FDA on the proteinuria and how much of that is actually written in stone per se? I'm just curious about the impact to that alignment if the Travere AdCom doesn't support two-year traditional approval. On number two, a question on Pompe. I appreciate that revenue growth does look second-half weighted, but curious on what you hope to be the exit rate going into 2026 and on a continued acceleration in 2026. I guess, what additional new countries do you plan to launch in 2026? Thanks.

Bradley Campbell (President and CEO)

Great. Thank you for the questions. Maybe we'll go kind of in reverse order. For Pombiliti, exactly right, we'll see a continued benefit of that acceleration in the second half. I think, even with all the kind of new launch countries that Sébastien highlighted, I think even a further impact in Q4 as an example, which is also pretty typical anyway to what we've seen with Galafold as Q4 tends to be a strong quarter. As we go to next year, TBD as it relates to run rate going into next year, but it's exactly the question to ask. We'll know more as we go forward here. I would just say that we do think next year will be a higher absolute revenue growth than this year. We'll have a lot more color to say on that going forward.

In terms of FSGS, I'll start, but then I'll have Jeff take over that question. As part of our diligence and as a prerequisite of the deal, we had to see the FDA minutes from their Type C meeting, and we were very pleased with the feedback that FDA had given to Dimerix in particular that proteinuria could serve as the primary endpoint for that study. That was really important for us to see, and I think an exciting development for the community. Jeff, speak a little bit to what we've said publicly around what that may mean for the primary and then how we see Travere's AdCom as it relates to our program.

Jeff Castelli (Chief Development Officer)

Yeah, thanks, Brad. As Brad said, the feedback from FDA for the Dimerix program is quite clear in terms of suitability of proteinuria at two years as a primary endpoint with just supportive data from GFR as a secondary. That can be measured as a percent change, as responder thresholds, or meeting certain thresholds of proteinuria, and we expect that we'll do all of those. As it relates to the AdCom it's not surprising there's an AdCom for FILSPARI. It's sort of the first product going through with proteinuria as a potential primary. They had a complicated phase III where the technical GFR endpoint was missed, and now they're looking at proteinuria as an alternative way for approval. I think net-net, that'll be a great conversation to have at that AdCom around proteinuria.

I think there's a number of really important similarities and differences between FILSPARI and DMX-200 that you have to think about around the AdCom. Clearly, similarities are they're both targeting very similar FSGS population, sort of primary genetic FSGS with significant proteinuria, and they're both planning to use proteinuria as a primary endpoint. Other than that, there's a lot of differences. The MOAs are very different. They target different underlying pathologies, the hemodynamic side of things versus the inflammatory side with DMX-200. DMX-200 is going to have a prospectively defined proteinuria endpoint for phase III. Ultimately, the data on proteinuria and importantly on GFR might be different for the two products. We view a positive AdCom reinforces proteinuria as a suitable endpoint, in particular when it's prospectively defined. We would not view a FILSPARI approval as a downside at all.

If anything, that would help start to really prep the FSGS community for new treatments. Mechanistically, we think they're very differentiated products and would work best in different types of patients and ultimately could be synergistic together. A negative AdCom, similarly, we think will inform us about how advisors FDA are viewing proteinuria, GFR, and will help us position our data. A negative outcome could be due to specifics around that kind of complex FILSPARI dataset and not necessarily read through to DMX-200 and ultimately could even position us as first to market if that did not work for FILSPARI. We view the AdCom as a positive positive for us. We're really looking to be informed by it. Ultimately, we would hope that it's a positive AdCom and FILSPARI will get approved for FSGS patients, and then we can quickly behind it have DMX-200 addressing a different aspect.

Bradley Campbell (President and CEO)

Sorry, one last question. Dennis, you also asked about additional countries for next year. Australia and Canada are two big ones. Over time, we'll continue as we did with Galafold, going into Asia-Pacific, into LatAm, into more European countries. There is still quite a bit of geographic expansion for Pombiliti as well. Thanks so much for the questions.

Dennis Ding (Analyst)

Perfect. Thanks, guys.

Operator (participant)

One moment for our next question. Our next question comes from the line of Salveen Richter of Goldman Sachs. Your line is now open.

Mark Aleynick (Analyst)

Hey, this is Mark on for Salveen. Thanks so much for taking our question. On DMX-200, when can we expect the phase III data? Also, what is the bar for success here and what would be clinically relevant in FSGS?

Bradley Campbell (President and CEO)

Jeff, do you want to take those, just a reminder of the timelines and then the clinical meaningfulness and kind of what we think the success bar would be?

Jeff Castelli (Chief Development Officer)

Yeah, thanks for the question. As we said, enrollment is going extremely well in the phase III study. We're on track for last patient in, which are 286 adult patients by the end of the year. That would mean last patient out two years, which would be end of 2027. That would be the timeline with the top line full two-year data. What was the second part of that question?

Mark Aleynick (Analyst)

What would be clinically relevant and the bar for success?

Jeff Castelli (Chief Development Officer)

Clinically relevant. One thing we're really excited about is from a powering perspective, with the 286 patients, two years, the study is powered to show small changes in proteinuria, percent changes less than 10% between groups or responder thresholds less than 10%. It comes down to clinical meaningfulness. Frankly, you could make an argument that any improvement in protein is clinically meaningful. I think one of the better ways to show that is through responder analyses. It's well established. If you can get patients below certain thresholds, like below 3 grams per gram or below 1.5 or below 0.7, those really improve the outcomes on progression to end-stage renal disease. I think looking at those responder thresholds will be important at a kind of patient level.

Mark Aleynick (Analyst)

All right. Thank you.

Operator (participant)

One moment for our next question. Our next question comes from the line of Xuan Hui. Your line is now open. Xuan, your line is now open. At this time, I don't see any further questions. This concludes today's conference call. Have a great day.